Pepgen Inc. (NASDAQ: PEPG) has just received authorization from the UK Medicines & Healthcare Products Regulatory Agency (MHRA) to begin the Connect2-EDO51 phase 2 clinical trial for PGN-EDO51, a potential treatment for Duchenne Muscular Dystrophy (DMD).
The Connect2-EDO51 trial is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) study, set to enroll approximately 20 ambulatory and non-ambulatory boys and young men living with DMD amenable to exon 51-skipping, who are at least six years of age. The participants will receive seven doses of either PGN-EDO51 or placebo at approximately four-week intervals for 24 weeks. The trial will assess exon skipping, dystrophin production, and safety and tolerability, and all participants will have the opportunity to participate in an open-label extension.
PGN-EDO51, Pepgen’s lead clinical candidate for the treatment of DMD, uses the company’s proprietary enhanced delivery oligonucleotide (EDO) technology to target the root cause of the disease by skipping exon 51 of the dystrophin transcript. In preclinical studies, PGN-EDO51 exhibited higher levels of exon 51 skipping in non-human primates compared to a structurally equivalent molecule, potentially leading to higher levels of dystrophin production in patients. In a phase 1 single ascending dose study in healthy volunteers, PGN-EDO51 showed six times higher mean exon 51 skipping compared to a naked oligonucleotide following a single dose.
DMD is an x-linked recessive, progressive, muscle-wasting disease that predominantly affects boys, with an incidence rate of approximately one in every 3,500 to 5,000 male births. It is characterized by progressive muscle weakness, leading to the loss of the ability to walk, upper body function, cardiac issues, and breathing difficulties. Despite significant advances in treatments, current exon skipping therapies are limited by poor delivery to muscle tissue and have yet to establish meaningful clinical benefit for DMD patients.
Pepgen Inc. is a clinical-stage biotechnology company aiming to transform the treatment of severe neuromuscular and neurological diseases through its enhanced delivery oligonucleotide (EDO) platform, which leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics.
This announcement marks a significant milestone in Pepgen’s efforts to develop a potentially transformative therapy for patients living with DMD, and the company looks forward to the potential positive impact of the Connect2-EDO51 phase 2 clinical trial on the treatment landscape for this devastating disease.
Today the company's shares have moved 1.9% to a price of $16.94. For the full picture, make sure to review PepGen's 8-K report.
